Select Committee on Health Minutes of Evidence

Memorandum by CancerBACUP (NC 26)


  CancerBACUP is the leading national charity providing information and support to people affected by cancer. The charity's specialist cancer nurses answer 65,000 enquiries a year from patients and carers on all aspects of cancer and its treatment. CancerBACUP's services include a telephone helpline, a wide range of booklets and factsheets, an interactive website and a network of local information centres. In addition to providing information and support, CancerBACUP works to promote patient-centred services and equitable access to effective treatments.

  CancerBACUP welcomed the creation of the National Institute for Clinical Excellence (NICE), in the belief that national guidance on the use of particular treatments and guidelines on the management of specific diseases would improve the quality of patient care and end unacceptable geographical variations in access to treatment. We welcome the opportunity to contribute as stakeholders to NICE technology appraisals and guideline development.

  This memorandum will focus on technology appraisals, as this is the aspect of NICE's work with which we are most familiar. Cancer treatments have featured prominently in NICE's work programme, and CancerBACUP has contributed to 11 separate appraisals to date. We have had a unique opportunity to present the patient's perspective to NICE, and as a result have acquired a broad insight into how NICE works and how it fulfils its remit of ensuring that patients have faster access to modern treatment.


  To date NICE has had a positive impact on the treatment and care received by people with cancer. However, we have a number of concerns relating to the transparency of the appraisal process; the absence of adequate data on quality of life issues; delays in issuing guidance which result in persistent problems with access to treatment while appraisals are taking place; and the inconsistency with which NICE guidance has been implemented to date. We also consider that the NICE process cannot in itself resolve the wider issue of ensuring sufficient funding for cancer services.


  1.  Greater transparency is needed on how particular treatments are selected for appraisal by NICE, how the Appraisal Committee reaches the decisions that determine the guidance it issues to the NHS and how the Committee evaluates different types of evidence.

  2.  To help inform NICE's decision-making, systematic research should be undertaken on patient priorities and issues relating to quality of life. NICE currently relies on patient organisations to provide information that should be collected systematically within clinical trials or as part of the NHS R&D programme.

  3.  The appraisal process should be faster, with shorter gaps between each stage of the process. In particular, if an appeal is upheld, the guidance should be redrafted immediately, rather than allowing months to elapse.

  4.  The Secretary of State should provide clearer guidance on whether particular treatments should be available to NHS patients during the appraisal process.

  5.  Implementation of NICE guidance should be monitored by the Department of Health, to ensure that NICE fulfils its remit of promoting faster access to treatment. This information should be made public.

1.  Transparency of the appraisal process

  1.1  The clarity and credibility of the guidance NICE issues to the NHS would be enhanced if the appraisal process were more transparent. A fully transparent process is vital also to ensure that the resulting guidance is locally owned and therefore effectively implemented.

  1.2  Organisations with an interest in the technologies referred to NICE for appraisal are invited to become stakeholders in the appraisal process. As a stakeholder, CancerBACUP has contributed to the process by providing written evidence to NICE, attending part of the Appraisal Committee's initial discussion of various technologies, responding to draft guidance produced by the Committee, and appealing against the Committee's final decisions on two occasions.

  1.3  Patients and carers have an interest in which treatments are referred to NICE for appraisal, and which are excluded. However, it is only once a particular technology has been referred to NICE by the Department of Health or the National Assembly for Wales that outside organisations are invited to contribute. At present it is not clear why particular treatments are referred and others are not. Some treatments NICE appraises are newly licensed, but others that are referred for appraisal have been available and in use by the NHS for a considerable time. The process by which technologies are selected for referral to NICE should become more open and transparent, so that a broader range of individuals and organisations have the opportunity to influence NICE's agenda and thereby influence national guidance on the use of NHS resources.

  1.4  Greater transparency is needed from NICE on the criteria used by the Appraisal Committee in reaching their decisions. For each appraisal, NICE commissions an independent assessment report undertaken by a health technology assessment centre. It should always be possible to see the connection between the conclusions drawn in the Assessment Report commissioned by NICE for each appraisal and the final guidance issued by the Committee. It would be helpful, not only for stakeholders involved in the process but also for health professionals tasked with implementing NICE guidance, if the guidance document stated explicitly which piece of evidence influenced each decision.

  1.5  Transparency is also needed in the process by which expert medical advisers are selected to advise the Appraisal Committee on particular appraisals. These advisers appear to play a crucial role in shaping the Committee's views, but it is not clear how the decision is taken on which experts should be invited. NICE has recently indicated that stakeholders can nominate expert advisers, but the process remains ad hoc, with some appraisals advised by two or three specialists in the field and others by only one.

  1.6  It would be helpful if the Committee provided stakeholders with detailed information about how the evidence is evaluated. Some organisations, including CancerBACUP, have been invited to contribute to a number of appraisals without having a clear idea of what sort of evidence is useful to the Committee. Given that many patient organisations invest considerable resources in contributing to NICE appraisals, it is reasonable to expect some feedback from NICE on whether, and in what way, the patient's perspective influences the Committee's decisions.

  1.7  Another area in which greater transparency would be welcomed by stakeholders is in definitions of cost-effectiveness used by NICE. The Appraisal Committee's remit includes assessing cost as well as clinical effectiveness. It is therefore important to understand the basis on which cost-effectiveness decisions are made. It is generally believed that NICE applies a cost-per-QALY[22] threshold of £30,000. However, this figure does not appear to be consistently applied and has not been made available to stakeholders and the wider public. NICE needs to clarify whether this threshold exists and, if so, how it was determined.

  1.8  Related to transparency is the need to ensure that appraisal guidance sets out explicitly what NICE intends, in order to avoid a situation where local funders interpret guidance too narrowly. For example, if NICE recommends that a particular treatment should not be "routinely used", the guidance either needs to state clearly in what circumstances the treatment could be considered, or it should state that clinicians should have the option to prescribe it. If it does not do this, local funders could decline to fund the treatment in any circumstances.

2.  Research on patients' priorities and quality of life

  2.1  If NICE is to provide guidance that is clear and credible - not only to the NHS which has to implement it but to patients who will be affected by it—it needs to base this guidance on high-quality information that accurately reflects the views and experiences of patients. This information is not always readily available.

  2.2  Most of the cancer treatments referred to NICE for appraisal offer significant benefits to patients in terms of quality of life, and many also offer additional survival time for people with advanced cancer. However, for people with advanced cancer, quality of life—by which we mean the extent to which people with cancer are able to continue with their usual activities free of pain and other symptoms associated with the disease—is often the most important objective and may matter as much if not more than extra weeks or months of life.

  2.3  Guidance issued by NICE suggests that the Appraisal Committee considers quality of life to be an important factor in determining which treatments should be recommended for use by the NHS. However, the experience of cancer appraisals has highlighted the absence of quality of life data in many clinical trials. The Committee does not always have available the evidence it needs to enable it to issue a positive recommendation to the NHS.

  2.4  One option, in the absence of suitable data from phase III randomised controlled trials (RCTs), would be for NICE to give more weight to other types of evidence. At present, NICE is reluctant to approve treatments that are not reinforced by data from RCTs. However, it is important that the Appraisal Committee acknowledges that it may be unethical to ask patients to take part in randomised trials if the drug being trialled is the only treatment available for a particular condition. For many types of advanced cancer, the only alternative to a new drug is best supportive care. Patients should have the opportunity to receive treatments that offer them extra survival time or a better quality of life.

  2.5  A more satisfactory long-term option is to require clinical trials to be designed to take account of quality of life. Much of the information that would be useful to NICE in formulating its guidance is not available, as clinical trials do not routinely collect data on the impact of a particular treatment on a patient's quality of life. In future we would hope to see cancer trials designed to collect quality of life data that both reflects the patient's true experience and satisfies NICE's need for systematic information on how particular treatments affect this experience.

  2.6  There may also be a role for the NHS R&D programme in conducting research on the patient's perspective on treatments for advanced cancer and other diseases, and on quality of life issues more broadly. NICE says that it genuinely wants the patient's perspective and sees this as central to the appraisal process.[23] If this is the case, NICE may wish to consider commissioning independent research on patients' views and experiences at the same time as it commissions the independent Assessment Report for each appraisal.

  2.7  A further option would be for NICE to ask the independent health technology assessment centres that compile assessment reports to take account of submissions from stakeholders when summarising the evidence on a particular treatment. At present the centres only consider published data, and submissions from stakeholders, including patient organisations, are considered separately by the Appraisal Committee.

  2.8  In the absence of systematic data on quality of life, patient organisations such as CancerBACUP have sought to fill in the gaps by providing the Appraisal Committee with an insight into the views and experiences of patients. However, this is a limited approach. It is very difficult for patient organisations to conduct an independent scientific assessment of the impact of particular treatments on the patients who receive them. If NICE considers patient organisations to be the most appropriate source of such information, there should be a system of reimbursement for these bodies. Reimbursement could come directly from the Department of Health, if NICE does not have the necessary resources.

3.  Timeliness of guidance

  3.1  It is vital that NICE appraisals are completed as quickly and efficiently as possible, to minimise uncertainty for patients and ensure that patients genuinely have faster access to effective treatments.

  3.2  While some appraisals last longer than originally envisaged because of a need to consider further evidence, lengthy delays have occurred for other reasons. It is of particular concern that the time between an appeal being upheld and the guidance being redrafted can be so long. During this time no further evidence can be submitted and patients are often unable to receive treatment (see section 4 below).

4.  Access to treatment during the appraisal process

  4.1  NICE plays an important role in ending confusion on the use of the treatments it appraises and in promoting faster access to these treatments for patients. However, this positive impact is undermined by the long delays in issuing guidance on some treatments.

  4.2  One of the reasons NICE was created was to speed up patients' access to effective modern treatments.[24] There are widespread expectations, not least within Government, that NICE guidance will resolve the problem known as "postcode prescribing", whereby access to particular treatments depends on where patients live and the decisions made by local health commissioners.

  4.3  However, there is a real issue about what happens while an appraisal is underway. Individual appraisals can last for a year or more, and there is much anecdotal evidence that some local commissioners do not fund treatments being appraised by NICE while an appraisal is ongoing. Far from speeding up access to treatment, the fact that particular treatments are referred to NICE appears to sow doubt in the minds of local funders about whether a treatment is worth funding. Patients have contacted CancerBACUP's telephone helpline to say that their oncologist wishes to prescribe a particular treatment but cannot do so because the treatment in question is being appraised by NICE and funds are not available until the guidance has been issued. Examples of these treatments are trastuzumab for breast cancer and irinotecan for colorectal cancer. This situation has become known as "NICE blight" and is acknowledged by the Department of Health, which has recently issued guidance to the NHS that a treatment for chronic myeloid leukaemia should not be denied to patients on cost grounds while NICE completes its appraisal.

  4.4  In spite of guidance to the NHS from the Secretary of State that NICE guidance should not be preempted locally,[25] a number of local commissioners have made clear to clinicians that funds will not be released for specific treatments unless and until NICE has given the go-ahead that they should be used. In the case of cancer, this means that patients are being denied treatments that their doctors want to prescribe and that may offer them extra survival time and improved quality of life. People with advanced cancer are not in a position to wait for NICE to finalise its guidance, and until guidance is issued clinicians should be able to prescribe treatments that they believe to be in the best interests of their patients. It would be helpful if the Secretary of State clarified and strengthened his previous guidance to the NHS to this effect.

5.  Implementation of guidance by the NHS

  5.1  NICE will only make a real difference to patients if the guidance it issues is implemented effectively. While the Government's recent announcement that NICE guidance will become mandatory for health authorities and primary care trusts from January 2002 is welcome, effective implementation will not take place unless the guidance is locally owned throughout the NHS. Greater transparency and stronger data should help promote local ownership. In the meantime, the announcement that guidance will be mandatory should help ensure more equitable access.

  5.2  An independent survey of health authorities in England and Wales commissioned by CancerBACUP and carried out in October and November 2001 demonstrates why it is necessary that guidance becomes mandatory.[26] Our research found that local ownership of national guidance is still some way from being achieved in many areas. Health authorities expressed the view that compliance with NICE guidance risks distorting local priorities. Others suggested that NICE guidance raises expectations among patients which are difficult to fulfil.

  5.3  CancerBACUP's survey found that NICE guidance is not being implemented uniformly throughout the NHS. While some health authorities have set aside funds to ensure that NICE guidance is fully implemented, the majority have not. Fewer than half the health authorities in England and Wales have a policy for monitoring local compliance with NICE guidance, and most do not know whether all suitable patients are being offered treatments recommended by NICE.

  5.4  The survey also showed that it takes many months for NICE guidance to be fully implemented. While most health authorities are implementing NICE guidance on taxanes for breast and ovarian cancer issued in the summer of 2000, far fewer are implementing guidance issued more recently on treatments for other cancers. It is vital that the Secretary of State clarifies whether the NHS will be expected to implement guidance already issued as well as guidance issued after January 2002.

  5.5  It is now imperative that the Department of Health monitors effectively the extent to which local health commissioners implement and fund national guidance. Systematic monitoring is needed and the results should be placed in the public domain to enable patients and the wider public to see what impact NICE is having on the treatment and care patients receive in practice.

December 2001

22   Quality-Adjusted Life Year. Back

23   NICE, Guidance for patient/carer groups (June 2001). Back

24   NICE, Faster access to modern treatment (1999). Back

25   Department of Health, Health Service Circular 1999/176 (6 August 1999). Back

26   CancerBACUP, Implementing NICE guidance on treatments for cancer (December 2001)-see Appendix attached. Back

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